Phase 1 Study of Autologous Regulatory T Cell NP001 Therapy for Adults with Amyotrophic Lateral Sclerosis ALS Evaluating Safety and Tolerability with Intrathecal Injection

What this Trial Is About

Researchers are conducting an open-label, multi-center phase 1 clinical trial to assess the safety and tolerability of an investigational treatment using autologous human polyclonal regulatory T cells NP001 cell injection in patients with neurodegenerative diseases, specifically amyotrophic lateral sclerosis ALS. The study aims to evaluate safety outcomes and identify the maximum tolerated dose. This study is sponsored by Novabio Therapeutics and focuses on patients diagnosed by established ALS criteria. Participants will undergo an apheresis procedure to collect their cells, which are then processed outside the body to enrich regulatory T cells. The investigational NP001 cell injections will be given intrathecally at doses of 1x106, 1x107, and 1x108 cells, administered up to three times at four-week intervals on Days 1, 29, and 57. Following the treatment period, participants will be monitored for approximately 12 months to assess safety and efficacy. During the study, participants will be regularly evaluated for treatment-related adverse events and dosage tolerance over six months. Functional assessments using the ALS Functional Rating Scale-Revised and Rasch Overall ALS Disability Scale will be conducted over the course of one year. The study involves various clinical and laboratory tests to monitor health status, and participants will be followed closely to capture both safety and functional outcomes throughout the trial duration.

Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases